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Childhood medulloblastoma.

Maura Massimino1, Veronica Biassoni1, Lorenza Gandola1

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Childhood medulloblastoma survival has improved by 30% in 20 years. Ongoing trials aim to reduce radiation for low-risk WNT subgroup patients and intensify treatment for high-risk cases.

Keywords:
MedulloblastomaMolecular subgroupsPediatric brain tumoursPrognosisRehabilitation in brain tumoursTarget therapy

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Area of Science:

  • Pediatric Oncology
  • Neuro-oncology
  • Cancer Genomics

Background:

  • Medulloblastoma is a common pediatric nervous system tumor, representing 15-20% of cases.
  • Survival rates have significantly improved over the past two decades.
  • Patient stratification into risk groups is crucial for treatment decisions.

Purpose of the Study:

  • To outline current and planned clinical trials for medulloblastoma.
  • To investigate treatment de-escalation for low-risk patients.
  • To explore treatment intensification and targeted therapies for high-risk and relapsed medulloblastoma.

Main Methods:

  • Risk stratification based on post-surgical disease, dissemination, histology, and molecular markers (WNT subgroup, MYC status).
  • Clinical trial designs evaluating reduced craniospinal irradiation (CSI) and posterior fossa radiotherapy (RT) doses for low-risk WNT medulloblastoma.
  • Clinical trials assessing intensified chemotherapy (CT) and radiotherapy for high-risk disease.

Main Results:

  • The WNT subgroup and MYC status are key molecular features for risk stratification.
  • High-risk medulloblastoma includes disseminated disease, residual tumors, and specific histotypes (large cell, anaplastic), or MYC amplification.
  • Ongoing trials focus on tailoring treatment intensity based on risk stratification.

Conclusions:

  • Treatment strategies for medulloblastoma are evolving towards personalized approaches.
  • Reducing radiation therapy is being explored for low-risk WNT medulloblastoma.
  • Intensified therapy and targeted agents are critical for improving outcomes in high-risk and relapsed medulloblastoma.